MuSK-stimulatory antibody

MuSK-stimulatory antibody is an experimental monoclonal antibody agonist that targets muscle-specific kinase (MuSK), a receptor tyrosine kinase essential for the formation and maintenance of neuromuscular junctions (NMJs). Developed by Genentech in collaboration with researchers at New York University, the antibody mimics the function of agrin to activate MuSK signaling, thereby stabilizing the postsynaptic membrane and preventing the synaptic loss characteristic of neurodegenerative diseases. In preclinical models of amyotrophic lateral sclerosis (ALS), specifically SOD1 G93A mice, the antibody has demonstrated the ability to preserve nerve-muscle connections, improve diaphragm function, and extend survival. Current research is focused on engineering a human-compatible version of the antibody, as the initial murine-targeted version does not cross-react with human MuSK.

Other names

MuSK agonist antibodyMuSK-activating antibodyMuSK agonist mAb